Drug Approval | February 24, 2026
Ultragenyx bags FDA priority review for groundbreaking GSDIa gene therapy
If approved, DTX401 would be the first treatment to address the disease at its root cause
If approved, DTX401 would be the first treatment to address the disease at its root cause
The therapy, also known as UM171 Cell Therapy, recently received conditional marketing authorization from the European Commission
MYC, a master regulator of oncogenic transcription, drives multiple high-burden cancers but has long eluded direct pharmacologic control
The approval marks a major advance in targeted treatment for this rare, chronic autoimmune disease, offering twice-yearly maintenance dosing after two initial loading doses
Under the deal, Uniphar will serve as LYMPHIR’s exclusive distribution partner across designated Western and Eastern European territories
The acquisition brings Lilly a novel in vivo cell therapy platform built around engineered circular RNA and proprietary lipid nanoparticles
The paper highlights how conditions common across India—including anemia, inherited hemoglobin disorders, and glucose-6-phosphate dehydrogenase (G6PD) deficiency—can significantly distort HbA1c readings
Venglustat, a glucosylceramide synthase inhibitor (GCSi), works by reducing the abnormal buildup of sugar-and-fat molecules in cells and organs
N17350 is the company’s first-in-class tumor-directed therapeutic, designed to harness the ELANE pathway
The product delivers 100 percent of the Recommended Dietary Allowance (RDA) of three essential nutrients—Biotin, Vitamin C and Zinc
Subscribe To Our Newsletter & Stay Updated
_256_275.jpeg)
